This was corroborated using systemic administration of SMN2-targeting ASOs in a mild SMA mouse model (Bogdanik et al., 2015), and in SMA mice sub-optimally dosed with a small molecule SMN2 splice-modifying drug and subsequently re-treated with the same compound or AAV1-follistatin gene therapy (Feng et al., 2016). The gene discussed is SMN2; the disease is proximal spinal muscular atrophy.